The present disclosure pertains generally to chemically-modified oligonucleotides for use in research, diagnostics, and/or therapeutics.
Certain RNA molecules are known to include repeat regions consisting essentially of repeating units of 3-5 nucleotides. Depending on the particular gene, the repeat region of a normal wild-type RNA molecule may comprise from about 5 up to about 11,000 copies of the repeating unit. In certain instances, the number of such repeating units can become increased and the resulting expanded repeat-containing RNA molecule may be disruptive to the cell. Certain diseases can result.
Certain oligonucleotides having nucleobase sequences complementary to a repeat region of a target RNA have been described, for example U.S. Patent Ser. No. 61/302,454; PCT International Application No. PCT/US2011/024019.